Phase I/II open-label trial of intravenous allogeneic mesenchymal stromal cell therapy in adults with recessive dystrophic epidermolysis bullosa

Ellie Rashidghamat, Tendai Kadiyirire, Salma Ayis, Gabriela Petrof, Lu Liu, Venu Pullabhatla, Chrysanthi Ainali, Alyson Guy, Sophia Aristodemou, James R. McMillan, Linda Ozoemena, John Mee, Rashida Pramanik, Alka Saxena, Rosamund Nuamah, Emanuele de Rinaldis, Sonia Serrano, Clarisse Maurin, Magdalena Martinez-Queipo, Su M. LwinDusko Ilic, Anna Martinez, Francesco Dazzi, Ineke Slaper-Cortenbach, Kasper Westinga, Sabrina Zeddies, Marcel van den Broek, Alexandros Onoufriadis, Jemima E. Mellerio, John A. McGrath

研究成果: Article同行評審

59 引文 斯高帕斯(Scopus)

摘要

Background: Recessive dystrophic epidermolysis bullosa (RDEB) is a hereditary blistering disorder due to a lack of type VII collagen. At present, treatment is mainly supportive. Objectives: To determine whether intravenous allogeneic bone marrow–derived mesenchymal stromal/stem cells (BM-MSCs) are safe in RDEB adults and if the cells improve wound healing and quality of life. Methods: We conducted a prospective, phase I/II, open-label study recruiting 10 RDEB adults to receive 2 intravenous infusions of BM-MSCs (on day 0 and day 14; each dose 2-4 × 106 cells/kg). Results: BM-MSCs were well tolerated with no serious adverse events to 12 months. Regarding efficacy, there was a transient reduction in disease activity scores (8/10 subjects) and a significant reduction in itch. One individual showed a transient increase in type VII collagen. Limitations: Open-label trial with no placebo. Conclusions: MSC infusion is safe in RDEB adults and can have clinical benefits for at least 2 months.

原文English
頁(從 - 到)447-454
頁數8
期刊Journal of the American Academy of Dermatology
83
發行號2
DOIs
出版狀態Published - 2020 8月

All Science Journal Classification (ASJC) codes

  • 皮膚科

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